Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic diseases including myotonic dystrophy (DM1). Thus, ASO strategies for DM1 can abolish the toxic RNA gain-of-function mechanism caused by nuclear-retained mutant transcripts conta...
| Hauptverfasser: | , , , , , , , , , , , , , , , |
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| Format: | Journal article |
| Sprache: | English |
| Veröffentlicht: |
American Society for Clinical Investigation
2019
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